Bellicum Announces Data Presentation Demonstrating Improved Immune Recovery Following Haploidentical Stem Cell Transplant with BPX-501 in Children with Blood Cancers and Nonmalignant Diseases

ATLANTA, Dec. 09, 2017 -- Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, today announced results from a leading European transplant center participating in the BP-004 trial in children with blood cancers and nonmalignant disorders. Patients were treated with BPX-501 following an alpha/beta T cell and CD19+ B cell depleted haploidentical hematopoietic stem cell transplant (haplo-HSCT). Results demonstrated that donor BPX-501 cells infused after transplant expanded in vivo and persisted over time, contributing to improved immune recovery for patients in the study as compared to historical controls from the same transplant center. The data were reviewed in an oral presentation today during the 59th Annual Meeting of the American Society of Hematology (ASH).

According to study author and presenter Pietro Merli of Ospedale Pediatrico Bambino Gesù in Rome, “These data show that administering BPX-501 cells following a haplo-HSCT may result in improved immune recovery and infection control, addressing significant risks in children undergoing a stem cell transplant who do not have access to a matched donor. Adding BPX-501 to a haplo-HSCT has the potential to improve outcomes and to make the curative benefits of transplants available to more children with cancers and genetic blood diseases.”

Study Design and Highlights (Abstract #211)

Investigators evaluated immune recovery and outcomes of 112 pediatric patients who underwent a haplo-HSCT followed by treatment with BPX-501. Children in the study had acute leukemia (n=53), Primary Immune Deficiencies (n=26), erythroid disorders (n=17), Fanconi anemia (n=7), and other diseases (n=9). All patients were transplanted after depletion of donor alpha/beta T cells and CD19 B cells to prevent graft-versus-host disease (GvHD) and post-transplant lymphoproliferative disorders (PTLD). BPX-501 cells were scheduled to be infused approximately two weeks post-transplant.  

Results showed:

• BPX-501 cells infused after haplo-HSCT expand and persist in patients, potentially contributing to improved recovery of adaptive immunity.
• Peak expansion of BPX-501 cells is reached at nine months after infusion, and BPX-501 cells are consistently detected after two years.
• CMV infection is a main driver of BPX-501 cell expansion, suggesting that BPX-501 cells cooperate in clearing the viral infection.
• The overall pattern of immune recovery in 112 children studied may be improved when compared to patients who received a similar haplo-HSCT without BPX-501.

“Improved immune recovery and control over infections, as demonstrated in this study, may have a direct impact on treatment-related morbidity and mortality,” commented Rick Fair, Bellicum’s President & Chief Executive Officer. “We look forward to reporting on these and other outcomes from our ongoing BP-004 trial in 2018.” 

A copy of the ASH presentation will be made available in the “Investors & Media” section of the Company’s website.

About BPX-501
BPX-501 is an adjunct T cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe^® safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. This enables physicians to more safely perform stem cell transplants by administering BPX-501 engineered T cells to speed immune reconstitution, provide control over viral infections and enhance Graft-versus-leukemic effect without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

About Bellicum Pharmaceuticals
Bellicum is a clinical stage biopharmaceutical company focused on discovering and developing cellular immunotherapies for cancers and orphan inherited blood disorders. Bellicum is using its proprietary Chemical Induction of Dimerization (CID) technology platform to engineer and control components of the immune system. Bellicum is developing next-generation product candidates in some of the most important areas of cellular immunotherapy, including hematopoietic stem cell transplantation (HSCT), and CAR-T and TCR cell therapies. More information can be found at www.bellicum.com.

Forward-Looking Statement

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Bellicum may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our research and development activities relating to BPX-501, BPX-601, BPX-701, rimiducid, CaspaCIDe, iMC, dual switch, CAR-T and TCR programs; the effectiveness of BPX-501, BPX-601 and BPX-701, their possible ranges of application and potential curative effects and safety in the treatment of diseases, including as compared to other treatment options and competitive therapies; the timing and success of our clinical trials, including our BP-004 study and observational trials; the rate and progress of enrollment in our clinical trials for BPX-501, BPX-601 and BPX-701, including our planned registration trials for BPX-501 and rimiducid; the timing of regulatory filings for BPX-501 and rimiducid; our research and development activities relating to our GoCAR-T and GoTCR technologies, and our collaborations with academic institutions. Various factors may cause differences between Bellicum’s expectations and actual results as discussed in greater detail under the heading “Risk Factors” in Bellicum’s filings with the Securities and Exchange Commission, including without limitation our annual report on Form 10-K for the year ended December 31, 2016 and our report on Form 10-Q for the quarter ended March 31, 2017. Any forward-looking statements that Bellicum makes in this press release speak only as of the date of this press release. Bellicum assumes no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Investors:
Bellicum Pharmaceuticals, Inc.
Alan Musso, CFO
832-384-1116
[email protected] 

Media:
BMC Communications
Brad Miles
917-570-7340
[email protected]

or

BMC Communications
Amy Bonanno 
914-450-0349
[email protected]

Source: Bellicum Pharmaceuticals